New FDA Breakthrough-Drug Category - Implications for Patients

作者：Murray Barbara E^*

来源：New England Journal of Medicine, 2014, 371(1): 88-88.

DOI：10.1056/NEJMhle1311493

In July 2012, the FDA Safety and Innovation Act created the breakthrough therapy designation to expedite development of therapies for serious diseases when evidence suggests substantial superiority over existing options. The authors discuss implications of the new designation. U.S. pharmaceutical regulations are based on the principle that patients should not be exposed to new prescription drugs until their efficacy and safety have been shown. Since 1962, the Food and Drug Administration (FDA) and Congress have balanced the efficient review of investigational drugs with the need to withhold judgment until sufficient evidence is available to clarify the benefit-risk relationship. Misjudging these competing interests in either direction causes important problems. On the one hand, the evidentiary hurdles of the FDA are often criticized by pharmaceutical companies and patient advocacy groups for slowing access to promising therapies. On the other hand, ...

• 出版日期2014-7-3